A Study of Pexa-Vec (JX-594) Prior to Sorafenib to Treat Unresectable Primary Hepatocellular Carcinoma
July 12, 2026 updated by: Jennerex Biotherapeutics
A Phase 2 Open-Label Pilot Safety Study of JX-594 Administered by IV Infusion Followed by Intratumoral Injection Prior to Standard Sorafenib Treatment in Patients With Unresectable Primary Hepatocellular Carcinoma
The purpose of this pilot safety study is to evaluate the safety and tolerability of JX-594 (Pexa-Vec) administered intravenously and intratumorally prior to standard sorafenib therapy.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
This is a Phase 2, open-label, pilot safety study designed to evaluate the safety and tolerability of sequential therapy consisting of Pexa-Vec (JX-594) followed by standard sorafenib in patients with advanced, unresectable primary hepatocellular carcinoma (HCC).
Patients will receive an initial intravenous (IV) infusion of Pexa-Vec at a dose of 1 x 10^9 plaque-forming units (pfu) on Day 1. This is followed by two intratumoral (IT) injections of Pexa-Vec on Day 8 and Day 22, each at a total dose of 1 x 10^9 pfu divided among 1 to 5 hepatic tumors. Starting on Day 25 (approximately 3 days after the final regular dose of Pexa-Vec), patients will initiate standard oral sorafenib therapy twice daily. For patients with viable hepatic tumor tissue at Week 12, an optional additional IT dose of Pexa-Vec may be administered.
The primary objective of this study is to assess the safety and toxicity of this sequential regimen, determined by the incidence of treatment-related Grade 3 and Grade 4 adverse events (AEs) and treatment-related serious adverse events (SAEs). Secondary objectives include evaluating the disease control rate (DCR) at 12 weeks, overall survival (OS) time, and radiographic response rate based on modified RECIST (mRECIST 1.0) and/or Choi criteria.
Study Type
Interventional
Enrollment (Actual)
25
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Busan, South Korea
- Pusan National University Hospital
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Yangsan, South Korea
- Pusan National University Yangsan Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Histological confirmation or clinical/laboratory diagnosis of primary hepatocellular carcinoma (HCC)
- Cancer is not surgically resectable for cure
- Child Pugh A or B
- Performance Score: KPS score of ≥ 70
- Platelet count ≥ 50,000 plts/mm3
- Total bilirubin ≤ 2.5 x ULN
- AST, ALT < 5.0 x ULN
- Acceptable coagulation status: INR ≤ 1.5 x ULN
- Acceptable kidney function: Serum creatinine < 2.0 mg/dL
- Sorafenib naive or refractory to sorafenib therapy Tumor Status: At least one intrahepatic tumor, and at least ≥50% of the total intrahepatic viable tumor mass, measurable by CT and injectable under imaging-guidance (note: injected and/or viable tumors must be previously untreated or ≥20% increase in size since preceding local-regional treatment).
Exclusion Criteria:
- Known contraindications to sorafenib
- Pregnant or nursing an infant
- Significant immunodeficiency due to underlying illness (e.g. hematological malignancies, congenital immunodeficiencies and/or HIV infection/AIDS) and/or medication (e.g. high-dose systemic corticosteroids)
- History of exfoliative skin condition (e.g. severe eczema, ectopic dermatitis, or similar skin disorder) that at some stage has required systemic therapy
- Clinically significant and/or rapidly accumulating ascites, peri-cardial and/or pleural effusions
- Severe or unstable cardiac disease
- Current, known CNS malignancy
- Use of anti-platelet or anti-coagulation medication
- Use of the following anti-viral agents: ribavirin, adefovir, cidofovir (within 7 days prior to the first treatment), and PEG-IFN (within 14 days prior to the first treatment).
- Patients with household contacts who meet any of these criteria unless alternate living arrangements can be made during the patient's active dosing period and for 7 days following the last dose of study medication:
- Pregnant or nursing an infant
- Children < 12 months old
- History of exfoliative skin condition that at some stage has required systemic therapy
- Significant immunodeficiency due to underlying illness (e.g. HIV/AIDS) and/or medication
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
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Experimental: JX-594 followed by sorafenib
1e9 pfu (plaque-forming units) total JX-594 dose on each of up to four (4) JX-594 treatment days.
Sorafenib is initiated after 3 JX-594 treatments and briefly interrupted if an optional 4th JX-594 treatment is given.
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Patients will receive a total dose of 1e9 pfu per treatment starting with one IV dose on Day 1 and injected intratumorally in 1-5 intrahepatic tumors on Day 8 and 22.
An optional maintenance JX-594 dose may be given intratumorally at Week 12.
Other Names:
Starting on Day 25 (3 days after the final JX-594 dose), patients will initiate oral sorafenib therapy twice daily according to standard approved guidelines.
Sorafenib therapy is briefly interrupted if an optional Week 12 JX-594 dose is given.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Determine safety and tolerability of intravenous infusion of JX-594 followed by intratumoral injections with JX-594 prior to standard sorafenib therapy
Time Frame: Safety evaluations through 28 days after last dose of JX-594
|
Adverse events will be collected and assessed to assess safety and tolerability through 28 days after last dose of JX-594 (or until all events considered probably or possibly related to JX-594 have resolved, stabilized, or returned to baseline status).
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Safety evaluations through 28 days after last dose of JX-594
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Determine Disease Control Rate (DCR) at 12 weeks
Time Frame: Disease control and response assessment at 12 weeks from first JX-594 dose
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DCR: confirmed complete response, partial response or stable disease based on modified RECIST and/or Choi response criteria
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Disease control and response assessment at 12 weeks from first JX-594 dose
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Determine radiographic response rate
Time Frame: Periodically throughout study participation (average of up to 1 year)
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Response rate evaluation based on modified RECIST and/or Choi response criteria
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Periodically throughout study participation (average of up to 1 year)
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Determine overall survival time
Time Frame: Ongoing (average of 1 year)
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Ongoing (average of 1 year)
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: David H Kirn, MD, Jennerex Biotherapeutics
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
May 26, 2009
Primary Completion (Actual)
September 27, 2012
Study Completion (Actual)
September 27, 2012
Study Registration Dates
First Submitted
July 26, 2010
First Submitted That Met QC Criteria
July 27, 2010
First Posted (Estimated)
July 28, 2010
Study Record Updates
Last Update Posted (Actual)
July 14, 2026
Last Update Submitted That Met QC Criteria
July 12, 2026
Last Verified
July 1, 2026
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Neoplasms by Site
- Neoplasms
- Infections
- Virus Diseases
- Neoplasms by Histologic Type
- Digestive System Neoplasms
- Digestive System Diseases
- Liver Diseases
- Neoplasms, Glandular and Epithelial
- Adenocarcinoma
- Poxviridae Infections
- DNA Virus Infections
- Carcinoma
- Carcinoma, Hepatocellular
- Liver Neoplasms
- Vaccinia
- Organic Chemicals
- Pyridines
- Heterocyclic Compounds, 1-Ring
- Heterocyclic Compounds
- Hydrocarbons
- Hydrocarbons, Cyclic
- Hydrocarbons, Aromatic
- Amides
- Benzene Derivatives
- Urea
- Acids, Heterocyclic
- Phenylurea Compounds
- Niacinamide
- Nicotinic Acids
- Sorafenib
Other Study ID Numbers
- JX594-HEP016
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
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